Of Interest – Focus on Rhabdohttp://focusonrhabdo.org
Advocacy, Resources & Information on RhabdomyosarcomaMon, 18 Sep 2017 18:22:42 +0000en-UShourly1https://wordpress.org/?v=4.8.260551081Caring at home for a child with cancer can leave family members with risky taskshttp://focusonrhabdo.org/caring-at-home-for-a-child-with-cancer-can-leave-family-members-with-risky-tasks/
http://focusonrhabdo.org/caring-at-home-for-a-child-with-cancer-can-leave-family-members-with-risky-tasks/#respondSat, 16 Sep 2017 14:41:21 +0000http://focusonrhabdo.org/?p=3175

Caring at home for a child with cancer can leave family members with risky tasks

Dan and Megan Kelley with daughter Bridget, who has leukemia. The girl has avoided infection since returning home in December. (Melissa Bailey/Kaiser Health News)

Angela Cooper arrived home from work to discover that her daughter’s temperature had spiked to 102 degrees, a sign that the teenager, who has cancer, had a potentially deadly bloodstream infection. As Cooper rushed her daughter to the hospital, her mind raced: Had she done something to cause the infection?Cooper, who works at a Chevy dealership in Iowa, has no medical background. She is one of thousands of parents who perform a daunting medical task at home — caring for a child’s catheter, called a central line, that is inserted in the arm or torso to make it easier to draw blood or administer drugs.

Central lines, standard for children with cancer, lead directly to a large vein near the heart. They allow patients with cancer and other conditions to leave the hospital and receive antibiotics, liquid nutrition and even chemotherapy at home. But families must perform daily maintenance that, if done incorrectly, can lead to blood clots, infections and even death.

As more medical care shifts from hospital to home, families take on more complex, risky medical tasks for their loved ones. But hospitals have not done enough to help these families, said Amy Billett, director of quality and safety at the cancer and blood disorders center at the ­Dana-Farber Cancer Institute/Boston Children’s Hospital.

“The patient-safety movement has almost fully focused all of its energy and efforts on what happens in the hospital,” she said. That’s partly because the federal government does not require anyone to monitor infections that patients get at home.

Even at the well-resourced, Harvard-affiliated cancer center, parents told Billett in a survey that they did not get enough training and did not have full confidence in their ability to care for their child at home.

The center was overwhelming parents by waiting until the last minute to inundate them with instructions — some of them contradictory — on what to do at home, Billett said.

An external central line, which has an end that lies outside the body, must be cleaned every day. Caregivers have to scrub the hub at the end of the line for 15 seconds, then flush it with a syringe full of saline or anticoagulant.

If caregivers don’t scrub properly, they can flush bacteria into the tube, and — whoosh — the bacteria enter a major vein close to the heart, Billett said. One father, noting that the hub looked dirty, scrubbed it with a pencil eraser, sending three types of bacteria into his child’s bloodstream, she said.

Monitoring infections

Learning the cleaning steps was “very nerve-racking,” recalled Cooper, whose 18-year-old daughter, Jaycee Gray, has had a central line since April to receive treatment for a rare type of blood cancer.

“You can scrub and scrub and scrub, and it doesn’t feel like it’s clean enough,” she said. Parents must keep track of other rules, too, such as covering up the central line before the child gets into the shower and changing the dressing if it gets dirty or wet.

But researchers recently discovered that more children with central lines are getting bloodstream infections at home: In a three-year study of children with cancer and blood disorders at 15 hospitals, 716 such infections took place outside the hospital, compared with 397 inpatient infections. This was partly because children with central lines spend much more time outside hospitals than inside them.

These hospitals belong to a national collaborative of 20 pediatric cancer centers that aims to train families, visiting nurses and clinic staff on how to handle central lines.

At one of the hospitals, Johns Hopkins in Baltimore, researchers discovered that patients as young as 8 were cleaning their own central lines at home, even though the hospital had designed its training materials for adults.

Cooper said that when her daughter developed the fever in July, she immediately started wondering if she were to blame: “It’s really hard,” she said. “I don’t want to put her in the hospital.”

When doctors confirmed that Jaycee had a bloodstream infection, Cooper asked them what caused it. Days later, after interviews and tests, no one knew for sure.

Jaycee was transferred to Children’s Hospital & Medical Center in Omaha, one of the other hospitals in the collaborative, where nurse Amanda Willits works with families to identify the likely causes of infections and to practice safe techniques. Willits said the bacteria probably came through the skin, but there is no sign that Cooper was to blame, and Cooper demonstrated her line-care technique perfectly.

Jaycee spent four days in an isolated room at the hospital. Doctors warned her that if the bacteria had colonized the plastic of her central line, she might have to go through surgery to have it removed and replaced.

As it turned out, Jaycee didn’t need surgery; she recovered with antibiotics.

In that study, pediatric oncologist Chris Wong Quiles of Dana-Farber/Boston Children’s tackled basic questions that researchers don’t have national data on: When patients get these infections at home, what happens to them, what does it cost and how often do they die?

Wong Quiles found that in 15 percent of cases, children ended up in the intensive care unit. Four children died. Their median hospital stay was six days, and their median age was 3.

These episodes also cost a lot. Wong Quiles found that median hospital charges were $37,000 per infection. That’s not counting professional fees from hospital staff; the cost of going home with antibiotics and possibly nursing care; or the cost to families of losing days of work to be at the hospital with their kids.

The ‘checklist engineer’

In Boston, Billett and Wong Quiles have enlisted extra staff and resources to try to help parents. The hospital hired what is called a “checklist engineer” to clean up inconsistent messaging and created family-focused videos, flip charts and pocket-size brochures about handling central lines.

Now, patients and families start learning central-line care five to 10 days before discharge, instead of just one or two days, Billett said. Parents first practice on a dummy called Chester Chest, then demonstrate their skills on their child.

Even after this training, bringing a child with cancer out of the hospital felt scary, said Megan Kelley, whose 8-year-old daughter, Bridget, is being treated there for leukemia.

“It felt like bringing a newborn baby home — we’ve never done this before,” said Kelley, who lives in Quincy, Mass., with her husband, Dan, and their three daughters.

Bridget and her family have managed to avoid infection since she was first discharged in December.

Along the way, the family got support and was spot-checked: The hospital keeps track of who was trained and that person’s skill level, and sends a nurse home to see how the caregiver handles the line.

This approach to patient safety — helping families at home through standardized learning tools, hands-on training and tracking skill development — could have broad applications for caregivers of patients young and old, Billett said.

These infections “can exact such a harsh toll on some of our most vulnerable patients,” said Michael Rinke, who led that research and now works at Montefiore Medical Center in New York. “Preventing even one of these can help a kid have an important out-of-hospital time, and have an important being-a-kid experience.”

— Kaiser Health News

Kaiser Health News, a nonprofit health newsroom whose articles appear nationwide, is an editorially independent part of the Kaiser Family Foundation.

Imagine an image of a sailboat atop blue water and surrounded by blue sky, he said.

Then imagine the pieces of that puzzle mixed with the pieces of 10 identical puzzles.

Now, try to put just one of them together.

That, Wilson said, is much like what happens when a scientist pulls data from the DNA of a single person’s genome.

Wilson is the executive director of the Institute for Genomic Medicine at Nationwide Children’s Hospital in Columbus. He opened his labs last week to give hospital employees and others a peek at the machines and researchers who are taking the first steps to help find new cancer treatments, reveal rare diseases and change the way medicine is practiced.

“There’s a whole bunch of cool stuff going on here,” Wilson said. “A lot of what we’re doing is very cutting edge.”

Among the machines he showed off was a sequencer, an instrument that saves scientists from having to dig through all those puzzle pieces.

The $750,000 machine reads DNA that is obtained from a patient’s tissue sample and placed on a “flow cell” that looks much like a microscope slide. The machine pulls information from the DNA, thereby sequencing the patient’s genome — a genome is the complete set of genes or genetic material in a cell or organism.

The sequencing runs for three or four days, sequencing the genomes of four to 16 samples at a time.

“The heart of the operation is instruments like this,” Wilson said. Without them, it would take an army of researchers years and cost $1 billion to read a single genome. At Nationwide Children’s, hundreds of genomes are sequenced every year, at a cost of about $15,000 each.

But that’s just the start. After data are pulled from DNA, the more than 20,000 genes must be analyzed to look for mutations. That’s done by researchers who sit in front of computer screens in the institute’s “dry labs.”

Once analyzed, the information is placed in the hands of other researchers or clinicians who can look for ways to treat specific mutations.

For example, the genomes of eight children with cancerous brain tumors were sequenced this summer, Wilson said. Seven of them received more information about their tumors, with six being given a new chance, taking drugs already found in the pharmacy but previously not used for their conditions.

“Genomics can really allow us to zoom in on exactly what’s gone wrong and maybe come up with a better treatment for the kids that we see,” Wilson said.

That’s called precision medicine, when clinicians tailor care to a patient instead of using a one-size-fits-all approach to cancers and other diseases.

The scale of the institute, and the level of expertise of Wilson and institute co-director Elaine Mardis, make it comparable to only a handful of such centers in the world, said Dr. Sameek Roychowdhury, an assistant professor of medical oncology and a research specialist in genomics and tumor DNA sequencing at Ohio State University’s Wexner Medical Center, which collaborates with Nationwide Children’s.

The institute will make Columbus a leader in genomics and precision medicine, he said, and allow it to attract more grant funding and experts in the field.

“All of these things are going to be affected by this institute,” Roychowdhury said. “It’s really going to be a magnet for change.”

The technology, he noted, is reshaping medicine. It leads to new ways of understanding disease and changes the way doctors diagnose illnesses, offer prognoses and treat patients. Clinicians who were stymied by rare diseases are re-diagnosing people after learning of specific genetic mutations.

For now, the main focus of the institute is cancer patients, and it reviews tissue samples not just from Nationwide Children’s patients but also from Wexner. But the analysis can be done on any patient. One example is a team of researchers that is trying to discover new ways to treat pediatric congenital heart defects.

“You can pretty much fill in the blank with your favorite childhood disease — that’s an opportunity for research,” Wilson said.

Next steps are to figure out how to do things faster so more genomes can be read and analyzed. Getting to more kids is the key, Wilson said.

A robotic machine that processes DNA recently was being tested by Natalie Bir, a technology development specialist.

A researcher can process up to 12 DNA samples a day, but the machine can do eight times that many.

“We can do 96 samples, which means we can put more samples on a sequencer and sequence more patients,” Bir said. “And physicians will be able to request more testing and, in the end, more kids will be able to be helped.”

The future is now, Bir said.

“Our genetic code says a lot about an individual, and I think there are a lot of unknowns about that, and our job is to try to figure out what all that means and help people survive in the end,” she said.

The institute is unique compared with many other places that do genomic testing in that it sequences cancer patients’ entire genome of more than 20,000 genes, instead of just 50 to 100 of the most likely culprits based on diagnoses, Wilson said.

“Using higher level methods ... is allowing us to find some things that are much less common and very novel and incredibly informative in terms of what the oncologists need to know to better treat the patients,” he said.

Although the more simple tests are covered by many insurance companies, the entire genome sequencing is not. The institute operates on philanthropic money.

It was opened in 2016 after the recruitment of Wilson and Mardis, who combined their expertise with that of groups already studying genomics at Nationwide Children’s.

“We’ve built that all into what we hope is going to be a real powerhouse for human genomics,” Wilson said.

Factors that increase your risk for a secondary cancer

Several factors can make you more likely to develop a secondary cancer. Some are under your control. Others aren't. Most of these risk factors have already been discussed in this report, but we repeat them here because it's important for you to discuss your risks with your doctor and find out what you can do to lower your odds of developing cancer again. Equally important, discuss how often you need to get screened, so you can catch any new cancers early.

Childhood cancer. If you developed cancer before age 15, you'll need to stay on top of your health in the years to come. Some childhood tumors are caused by inherited syndromes that contribute to a lifelong increased risk for cancer. For example, Li-Fraumeni syndrome can lead to sarcoma, leukemia, and brain and breast cancers. The treatments you received to combat childhood cancer can also make you more vulnerable to future malignancies.

Family history. When you have multiple close relatives who all developed a particular cancer, that's a very strong indication that your family carries a genetic susceptibility. Though you can't change your genes, you can get tested for genetic changes that are associated with increased cancer risk and—if you are at higher risk—be screened for those cancers and take other preventive measures.

Cancer treatment. Radiation, chemotherapy, and other cancer therapies, while necessary to cure your disease, can also trigger cellular changes that make you more vulnerable to a secondary cancer. Your doctor will make every possible effort to structure your initial treatment—for example, fine-tuning the drug and dose—to destroy the cancer, while minimizing your future cancer risks.

Age. The older you are, the higher your cancer risk. Each passing year brings more chronic conditions, more exposure to environmental factors that increase your risk, and a lower ability of cells to repair damage.

Lifestyle. Lifestyle is one cancer risk you can control. Many of the choices you make each day can influence—sometimes significantly—your chances of getting a future cancer. Here are a few things you can do to reduce your risks:

Patient Voices: Childhood Cancer

[COMMENT: Not Rhabdo specific but a very powerful message about childhood cancer. I cannot get the audio links into this post, so please visit the Original Article to listen to the audio. - Alan]

Cancer is the leading cause of disease-related death among children ages 15 and younger. Approximately 10,000 new cases are diagnosed every year, more than half of them leukemia or cancers of the central nervous system. How is treating cancer in children different from treating it in adults? How do children cope with a disease they can hardly understand? And what happens to a family after losing a child to cancer?

Still a Mom

Jaiden Ramirez, 7, Voice of mother, Toshia Ramirez, Houston

Play Audio

2:18

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Toshia Ramirez, a master-at-arms in the Navy, has two sons. One of them, Jaiden, has an inoperable tumor surrounding his brainstem. Physicians believe he has only a few months left to live.

In December, when his mother was stationed in Guam, Jaiden developed poor balance and difficulty speaking and writing. A few weeks later, doctors realized he had a brain tumor, and Jaiden and his family were transferred to Houston for medical care. Jaiden was thin and active before beginning chemotherapy. But the medication has made him bloated, and Jaiden is often tired and nauseated.

The Ramirezes are staying in a Ronald McDonald House in Houston while Jaiden gets medical treatment in a nearby hospital. All of the family’s belongings are still in Guam.

Ms. Ramirez has spoken with Jaiden about his cancer and the possibility that he will die. She prays for a miracle and hopes that a clinical trial that Jaiden has entered will extend his life long enough for a new treatment to emerge. In the meantime, Ms. Ramirez does her best to stay positive and enjoy every moment she has with her son.

A Boy’s Life, Restored

John Carey, 16, Colorado Springs

Play Audio

2:03

Kevin Moloney for The New York Times

Kevin Moloney for The New York Times

Kevin Moloney for The New York Times

Kevin Moloney for The New York Times

Kevin Moloney for The New York Times

John Carey found out that he had an advanced form of Hodgkin’s lymphoma at age 13, during a routine physical to join his school’s wrestling team.

John’s doctor first believed he had a heart murmur, but further tests showed a tumor in his chest that had begun to affect his heart. John immediately began chemotherapy.

Highly treatable, Hodgkin’s lymphoma generally responds to a few rounds of chemotherapy. But John had to have 11 rounds, a stem cell transplant and radiation treatment over the course of two years before his cancer was in remission. John now sees his doctor every three months to monitor his health and ensure that the cancer does not relapse.

John said that today he is happy to be able to hang out with his friends again and resume normal life.

Finding a New Voice Amid Loss

Max Mikulak, 7, Voice of Melissa Mikulak, San Diego

Play Audio

2:38

David Ahntholz for The New York Times

David Ahntholz for The New York Times

David Ahntholz for The New York Times

David Ahntholz for The New York Times

David Ahntholz for The New York Times

David Ahntholz for The New York Times

Melissa Mikulak, lost her son Max to neuroblastoma when he was 7.

At age 3, Max began frontline treatment for his cancer. He responded well to chemotherapy, although he suffered hearing loss. A year later, doctors said he had no evidence of disease.

Children with neuroblastoma have an 80 percent chance of relapse, and Max’s cancer came back when he was 5 years old. After a few clinical trials, all treatments stopped working.

After Max’s death, the Mikulaks created a table in their home in his memory. It holds some of his favorite toys and photos, as well as the urn that contains his ashes. Ms. Mikulak said that family members visit the table when they want to feel closer to Max. Bruce the Whale was Max’s favorite toy. The Mikulak family includes the toy in photos and brings it on trips as a way of keeping Max in their lives.

Ms. Mikulak said that helping her other children cope with the death of their brother has been challenging at times. Her daughter, Hannah, 11, sees a therapist, but Ms. Mikulak is still trying to figure out how to help Nick, 5, understand everything that’s happened.

After Max’s death, Ms. Mikulak and her husband, Andy, created a nonprofit organization, Max’s Ring of Fire, to help raise money for pediatric cancer.

Living With Cancer’s Long-Term Impact

Kayla Cooper, 25, Houston

Play Audio

2:06

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Michael Stravato for The New York Times

Kayla Cooper, a student at the University of Houston, was 15 months old when she was found to have neuroblastoma, a malignant cancer, on her right kidney and spinal cord.

Because of how the cancer grew, Ms. Cooper is paralyzed from the waist down and she relies on a wheelchair and other assistive devices to get around.

Three years ago, Ms. Cooper became pregnant with her daughter, Kimora. Because of the chemotherapy and radiation treatments Ms. Cooper went through as a baby, doctors were concerned about how her heart would respond to the rigors of pregnancy. They closely monitored Ms. Cooper, and her pregnancy was uneventful.

Ms. Cooper is in school, working toward a master’s degree in social work. She believes that her experiences as a cancer survivor have shaped her desire to help others.

Childhood cancer survivors are at a higher risk for developing a new cancer later in life. Ms. Cooper monitors her health with annual checkups and keeps in close contact with her doctors.

A Mother’s Never-Ending Worry

Sarah Goldberg, 13, Voice of Robin Goldberg, Westlake Village, Calif.

Play Audio

2:09

David Ahntholz for The New York Times

None

David Ahntholz for The New York Times

David Ahntholz for The New York Times

David Ahntholz for The New York Times

When Robin Goldberg’s daughter, Sarah, was born, she had a difficult time eating and did not gain weight as she should have. Her pediatrician called it “failure to thrive” but could not determine the cause.

After an X-ray to determine if a feeding tube was inserted properly, a radiologist found that Sarah, then 6 months old, had a large tumor in her abdomen. It was a mature teratoma, a benign tumor containing a variety of tissues, and it was surgically removed two days later.

Even after the tumor was removed, Sarah did not gain weight well. Her parents fed her constantly, but Sarah would frequently vomit. She was hospitalized for dehydration and confined to her home for five months to prevent infection. When Sarah turned 5, Ms. Goldberg finally felt like the worst was over.

As Sarah grew, however, she developed scoliosis, a curvature in her spine, which may have been caused by the tumor.

Sarah now wears a brace to keep her spine growing correctly. Ms. Goldberg, who did graduate work on cancer, said that her daughter’s cancer experience had changed her. “To me it was nothing but a science,” she said. “Then Sarah came along and put a lot of emotion to it.”

Patient Voices is an audio-visual series that tells the stories of people living with chronic illness. Patient Voices: Childhood Cancer was originally published in May 2011.

Summary

Retinoblastoma, a rare form of eye cancer, usually develops in early childhood. Human tumor cells implanted and grown in mice could offer researchers a new way to study the cancer. A stained sample of one such tumor, or orthotopic xenograft, is shown. Credit: Michael Clay, MD

Howard Hughes Medical Institute (HHMI) scientists have created an extensive resource for studying pediatric cancers, which they are sharing widely to help accelerate research.

Led by HHMI Investigator Michael Dyer at St. Jude Children’s Research Hospital, the team grew cells from patient tumors in laboratory mice, and created nearly 100 models of 12 types of pediatric cancer. The researchers implanted the tumor cells into their organ of origin in the mice and carefully characterized them to ensure clinical relevance. Now, Dyer and his colleagues are making samples freely available to the scientific community through the Childhood Solid Tumor Network. Researchers worldwide will also have access to data about the tumors’ sensitivity to drugs, molecular profiles, and other features.

“We want this data at the fingertips of clinicians, translational researchers, and basic scientists,” Dyer says. “It’s a great resource for people interested in moving new therapies forward.”

Dyer’s team has already used the models to identify a new drug combo that may benefit patients with recurrent rhabdomyosarcoma, an aggressive childhood cancer that begins in muscles or other soft tissue. The team describes the new models and reports its findings August 30, 2017, in the journal Nature.

According to the American Cancer Society, pediatric cancers represent less than one percent of all cancers diagnosed each year in the United States. Until now, scientists have had few resources available to study how they develop. Five years ago, Dyer and his colleagues set out to create laboratory models that would give researchers new tools to study these cancers. Their goal was to broadly represent the diversity of tumors that develop in children. “I knew I wanted to start with all pediatric solid tumors, not just pick one or two,” Dyer says.

An electron microscopy image of rhabdomyosarcoma, an aggressive childhood cancer that begins in muscles or other soft tissue, reveals the cellular interior. Credit: CTI-EM Core, St. Jude Children’s Research Hospital

Over the last five years, he and his colleagues obtained tumor samples from 168 patients, including tumors that arose when a patient’s disease recurred after initial treatment. Cells from the tumor samples, which represented 15 types of pediatric cancers, were injected into mice and allowed to grow.

A human tumor that is grown in a mouse or other animal is called a xenograft. It is most commonly implanted by injecting tumor cells just under the animal’s skin. Instead, Dyer’s team wanted to grow their xenograft tumors in the relevant tissues; these tumors are called orthotopic xenografts. The researchers they knew that a tumor’s development is influenced significantly by its microenvironment in the body.

They figured out how to get tumor cells to their tissues of origin, and then implanted each patient tumor sample into multiple mice. Not all the tumors grew in the animals, but the team was able to establish 97 patient-derived xenografts representing 12 types of pediatric tumors.

Dyer’s team then compared the molecular and cellular features of the mouse tumors to those of the patient tumors from which they were derived. Many of the mouse tumors retained the complex makeup of the patient tumors, the researchers found.

“We know that cancer isn’t a homogeneous population of tumor cells. It’s a mixture of different cells,” Dyer says. “For at least some of the patients, we’re able to capture that complexity.” This is important, because a tumor’s cellular composition can change dramatically after treatment, and the cells that persist largely determine whether a patient’s cancer recurs, he says. “With the xenografts, we can for the first time model this complexity in the laboratory.”

Once the models were established, Dyer’s team grew cells from 30 of the xenograft tumors in culture dishes and used them for large-scale drug screens. They determined each tumor’s sensitivity to 156 drugs, producing more than a half million data points.

The team discovered that the muscle cancer rhabdomyosarcoma is sensitive to a combination of three drugs, two of which – irinotecan and vincristine – are already widely used treatments for the disease. The third drug, AZD1775, is an inhibitor of the enzyme WEE1, a key regulator of cell cycle progression. Its safety when used in combination with irinotecan has recently been evaluated in pediatric patients in a phase I clinical trial.

In tests in mice with rhabdomyosarcoma xenografts, the three-drug combo had a greater effect on tumor size and growth than the standard drug regimen. “There was a dramatic response pretty much across the board for these aggressive rhabdomyosarcoma patient-derived tumor samples,” Dyer says. He is optimistic that the new drug combination will move into clinical trials quickly, and that it may bring real benefit to patients with this difficult-to-treat disease.

Dyer’s team has made the drug sensitivity data from the lab’s screens available in a free, easy-to-use online database. “The faster and easier the data is to use, the easier it is for people to test hypotheses,” he says.

Likewise, Dyer is eager for the research community to use the new xenografts. Cells from each patient-derived tumor have been preserved for future studies and the team has already distributed samples to more than 130 labs worldwide. All the data that Dyer’s team has collected with the models is available upon request including unpublished data. The team will continue to expand the resource, developing new xenografts and incorporating more tumors that represent rare subsets of pediatric cancers.

Park Ridge resident Suzie Tinagli listens as Dr. John Cunningham, chairman of the pediatrics department at University of Chicago Medicine Comer Children’s Hospital, talks with her son Sam earlier in August before he headed to college. The 18-year-old has been in remission from acute lymphoblastic leukemia since he took part in a clinical trial for a new therapy, which gained FDA approval Aug. 30, 2017. (University of Chicago Medicine)

[COMMENT: So nice to see a new treatment being prepped for pediatric use so quickly. Hopefully this is a sign of things to come for the Rhabdo community - Alan]

The treatment, which uses a patient's own modified cells to battle a form of acute lymphoblastic leukemia, is the first gene therapy approved for use in the U.S. Acute lymphoblastic leukemia is the most common childhood cancer.

Its $475,000 price tag is drawing jeers from some advocacy groups, but many are also excited about the treatment's potential to save lives.

Here's how the CAR-T cell therapy works: Immune cells called T-cells are collected from a patient's blood and genetically engineered to fight the leukemia. Those supercharged T-cells are then put back into the patient's body, where they kill the cancer cells. The therapy came out of a collaboration between Switzerland-based Novartis and the University of Pennsylvania.

The treatment is intended to help the roughly 15 to 20 percent of children and young adults with the disease who don't respond to initial treatment or whose cancer returns after initial treatment, according to the FDA.

Until now, children with those tougher cases of the disease had low survival rates, said Dr. John Cunningham, chairman of the department of pediatrics at University of Chicago Medicine Comer Children's Hospital. In a clinical trial, more than 80 percent of patients who underwent CAR-T cell therapy went into remission, according to the FDA.

"In my 30 years of being an oncologist, that's the most dramatic difference I've ever seen," Cunningham said. "I've never seen something so spectacular."

University of Chicago Medicine has been participating in a clinical trial of the treatment, which will be marketed under the brand name Kymriah.

More than 3,000 people under age 20 are diagnosed with acute lymphoblastic leukemia each year, according to the National Cancer Institute, and the new treatment is designed for the most common form of it.

For now, only a select group of hospitals across the country will be allowed to offer Kymriah commercially because of its potentially severe, life-threatening side effects, including high fever, flu-like symptoms, infections and low blood pressure. The FDA is requiring hospitals and clinics that offer the therapy to be specially certified and have another newly approved drug on hand to treat some of those side effects.

Representatives from University of Chicago Medicine and Lurie Children's Hospital confirmed Wednesday they are working with Novartis to be part of a network of hospitals offering the therapy commercially.

Cunningham said University of Chicago Medicine could potentially begin offering the treatment to patients within weeks. Lurie could also begin offering it in coming weeks, said Dr. Reggie Duerst, clinical director of Lurie's stem cell transplant program.

Novartis spokeswoman Dana Cooper said in an email Wednesday she could not provide the names of hospitals that will offer the therapy commercially in Chicago. Novartis is aiming to have at least 20 medical centers certified to offer the treatment within a month, she said. The company hopes to have 32 centers certified by the end of the year.

Novartis said Wednesday it will charge $475,000 for the therapy, and it's working with insurers to make sure they provide coverage for patients. She said the price is below current standards of care, such as the cost of some stem cell transplants.

Novartis believes the $475,000 price will "support sustainability of the health care system and patient access while allowing a return for Novartis on our investment," she said.

But the Campaign for Sustainable Rx Pricing criticized the price tag. "While science behind Kymriah is revolutionary, the business decision to price it at nearly half of a million dollars per treatment is not," it said in a statement. "Kymriah's price tag is simply a continuation of the pattern of sky-high launch prices that spins further out of control each year."

The group did, however, say it's excited about the therapy's potential to save lives.

Some patients, such as Sam Tinaglia, of Park Ridge, have already seen the difference it can make.

Tinaglia was diagnosed with the disease when he was 5 years old, but he relapsed after initial treatment. He endured about eight years of chemotherapy and a bone marrow transplant before Cunningham, his doctor, helped him take part in a clinical trial of the treatment in Philadelphia in 2015.

Like many patients, he suffered severe side effects from the therapy. He had high fevers, memory loss and a seizure, said his mother, Suzie Tinaglia. But within about a week in the clinical trial, those symptoms cleared. He's been in remission ever since.

Sam, now 18, graduated from Maine South High School this year, and his parents dropped him off at the University of Illinois, Urbana-Champaign, for his freshman year about two weeks ago.

"We never thought he would even be able to go to college," Suzie Tinaglia said. "You just go back to your life and your body heals itself. It's really amazing."

Suzie Tinaglia said Novartis covered the costs of her son's treatment because he was part of a clinical trial.

Several other companies have also been conducting clinical trials for CAR-T cell therapies.

A study by the MGH Cancer Center has found that out-of-pocket health care costs can lead to financial problems for suvivors of childhood cancer. Credit: smartstock

Adult survivors of childhood cancer face an increased likelihood of financial difficulties related to out-of-pocket costs for their health care, compared with other adults. In a report published online in the Journal of Clinical Oncology, investigators from the Massachusetts General Hospital (MGH) Cancer Center also report that survivors of childhood cancer who pay higher out-of-pocket costs were more than eight times as likely to have trouble paying their medical bills than were either survivors not facing higher out-of-pocket costs or adults without a history of childhood cancer.

“Survivors who reported spending a higher percentage of their income on out-of-pocket medical costs were not only more likely to report financial burden, they also were at risk for undertaking behaviors potentially detrimental to their health in order to save money,” says MGH Cancer Center’s Ryan Nipp, lead and corresponding author. “While studies have identified associations between financial burden and patients’ treatment outcomes, quality of life, and even survival among adults with cancer, as far as we know, this is the first to report these associations in survivors of childhood cancer.”

Successful treatment of childhood cancers has led to an increase in the number of adult survivors, but studies also have reported that these individuals are at elevated risk for chronic health conditions, such as heart disease, kidney impairment, and secondary cancers. Many health care plans have increased the out-of-pocket costs for which patients are responsible through cost-sharing measures such as increased deductibles, co-payments, or co-insurance. The current study was designed to investigate the extent to which increased out-of-pocket health care costs pose a financial burden to survivors of childhood cancer, and the potential consequences.

The research team surveyed participants in the Childhood Cancer Survivor Study (CCSS), which enrolled adults who had been treated for childhood cancers between 1970 and 1986 along with a control group of siblings not affected by cancer. In 2011 and 2012, a group of CCSS participants — both survivors and siblings — was surveyed regarding the type of health insurance they had; out-of-pocket health care costs paid during the previous year; income, employment, and other sociodemographic information; and whether medical costs posed a financial burden and if so, the measures taken to deal with those financial pressures.

Complete responses — including information on income and out-of-pocket health care costs — were received from 580 childhood cancer survivors, who were an average of 30 years from their initial diagnosis, and 173 siblings. Ten percent of survivors were determined to have higher out-of-pocket health costs, defined as 10 percent or more of household income; less than 3 percent of their siblings faced higher out-of-pocket costs.

Survivors reporting higher out-of-pocket health care costs were likelier to have lower incomes and to report being hospitalized in the past year. To offset these financial burdens they reported skipping treatments, tests, or follow-up visits; postponing or delaying medical care; and taking smaller-than-prescribed doses of medication, among other measures. Compared both with survivors paying lower costs and with siblings, survivors paying higher out-of-pocket costs were likelier to worry about their ability to access health care and to think about declaring bankruptcy.

“A more comprehensive understanding of the relationship between high out-of-pocket medical costs and the adverse effects of increased financial burden on cancer survivors could be instrumental in helping us identify those at risk for higher costs to help us address their financial challenges and improve health outcomes,” said Nipp, an instructor in medicine at Harvard Medical School. “It could also help inform policy changes to help meet the unique needs of cancer survivors and improve our understanding of how both higher costs and resulting financial burden influence patients’ approach to their medical care and decision-making.”

The authors note that, since the study was conducted prior to the full implementation of the Affordable Care Act, there is no way of knowing how the ACA may have influenced high out-of-pocket costs and financial burden among survivors of childhood cancer, something that should be studied in future research. They also cite the need to study ways of incorporating financial discussions within survivorship clinics and developing programs that include elements such as financial services, patient navigators, and social workers to help patients plan for and deal with the economic challenges related to cancer and cancer care.

[COMMENT: A good idea. Perhaps this is something that each of could do in our respective state? - Alan]

AURORA, Colo. -- Gabriel Santistevan wants to put a daily reminder about childhood cancer right in front of thousands of Colorado motorists.

While he's going through his own fight with cancer, the Aurora teen has designed a unique way to help other families going through the same struggle.

He and his mom are petitioning the state for a custom license plate to support the Morgan Adams Foundation and Cops Fighting Cancer. The Aurora based group of police officers have supported Gabe and other families for 15 years.

Gabe was diagnosed with stage 4 brain cancer at 9 years old and his future was in doubt.

"He went through chemo, radiation he had a craniotomy to remove tumor,” said his mother Kathleen Santistevan. “And he's been struggling with health issues ever since he's now 13.”

"I thought that if I just keep fighting,” Gabe told FOX31. “And everybody a lot of people pray and I pray just the cancer would never come back."

For Gabe and his mom it's been an uphill struggle along with other families fighting the same fight.

"When this first started out, we were in the hospital and saw so many kids. There's an entire floor in Children's Hospital of kids fighting cancer. We had no idea," Kathleen said fighting back tears. "I didn't know that he was going to survive I didn't know what was going to happen."

To help raise money for research they came up with a license plate design featuring a gold ribbon that reads “Cure Childhood Cancer.”

Social isolation

Often young cancer survivors won't have met anyone else their age who's had the same experience.

It can be terrifying for everyone — including their friends and family — to think that someone so young can become so ill. No-one has the words to make sense of what's happening.

Serena (R) and her mum Jane (Supplied)

"I found it incredibly isolating. I didn't have anyone that I felt really understood," Serena says.

Not knowing how to reach out, young people often hide their true feelings and play the role of the 'model' cancer patient — trying to think positive, stay strong, and above all, fight their cancer.

For Serena, putting on a front included playing a comforting role for those closest to her.

Like when she told her distraught mother "everything happens for a reason" just moments after hearing her diagnosis.

Once she became a survivor, continuing with that coping mechanism meant it took her a long time to open up and get support for the inner emotional turmoil she was feeling.

The 'positives' can come slowly

Being back in the safety of home often isn't enough to stop cancer from dominating the psychology of a young survivor.

Even today, the smallest things can trigger Serena to go back to feeling how she did when she had cancer.

"I can go back into the story of myself as being kind of weak, and sick, and disempowered," she says.

Some survivors experience profound insecurity because they don't trust their body — they think it will let them down again, and worse still, the cancer will return.

People often tell survivors that they are "strong", "brave" and "inspirational". But these words can jar with someone who feels they had no choice but to deal with it.

Still, facing the fall-out of their cancer for years afterwards does forge a new kind of strength for many.

"I have incredible resilience now, as a person," Serena says.

"I've got a totally different perspective than I ever did before."

Serena, a few months into treatment, when she started to lose her hair (Supplied)

What most survivors come to realise is that the positives come because of the trauma they've been through, rather than instead of it.

Their new outlook on life is hard-won: through surgical scars and bald scalps; through missed school camps and work shifts; through a friend's silence on the other end of an SMS; through watching other kids on the ward die.

It's useful to know that the 'positives' can take a while to come, but not everyone will feel like a better person for having survived cancer.

"I've grown so much, and I feel so blessed," Serena says. "But the vulnerability [is] still part of me. Sometimes I really feel like that little girl who's got no eyebrows."

Joining a club you never wanted to join

Everyone is affected by cancer differently, and some people don't relate to the term 'survivor'.

But whatever their differences, many survivors find the antidote to their uncomfortable reactions is connecting with others in the same boat.

A year after she got the all-clear, Serena joined an online psychological support group called Recapture Life.

Together with other young survivors, she had video conversations with a psychologist to explore new ways of coping with life after cancer.

A video session with Recapture Life (Supplied: UNSW)

"It's hard to put into words, but you just can't underestimate the power of the innate understanding we shared because we'd been through the exact same thing," she says.

What works for some, may not work for others. But better support for young cancer survivors means acknowledging they're still living with challenges.

The end of treatment is just a stage in a long journey and it may bring up uncomfortable things they need permission to think about, feel, and explore.

For Serena, her online group gave her a safe space to process her cancer experience — and move towards recovery.

"When you're given the opportunity to be a little more vulnerable, and a little bit more open, that's when you can start to really transform through your experience," she says.

Dr Ursula Sansom-Daly is a clinical psychologist and researcher at the University of New South Wales. She is also one of RN's Top 5 Under 40 scientists.

Senate passes bill to improve cancer drugs for children

WASHINGTON — Until now, drug companies have beenfree to decide whether to pursue treatments for pediatric cancers as part of their work on adult cancers.

They won’t have much choice going forward.

The Senate on Thursday overwhelmingly passed legislation requiring the pharmaceutical industry to expend more resources on treatment for childhood cancers. The bill, part of a larger measure reauthorizing user fees imposed by the Food and Drug Administration, heads to President Trump for his expected signature.

Existing law directs companies to study the safety and efficacy of adult drugs on children unless the FDA gives them a waiver. Medicine developed to treat heart disease or diabates for adults, for example, must also be tested for its use on children.

But when it comes to cancer, advocates say the FDA has too much latitude to exempt the industry from studying and developing help for kids. Federal regulators often have not required companies that invest heavily in the four major cancers — breast, prostate, lung and colon — to research how the treatments they develop for those adult-oriented diseases might assist in addressing childhood cancers.

Over the past 20 years, the FDA has approved about 190 new cancer treatments for adults but only three for children, said Sen. Michael Bennet, a Colorado Democrat who co-sponsored the provision with Florida Republican Sen. Marco Rubio.

“That meant our kids continue to receive older treatment, some from the 1960s that often had harmful side effects and consequences that can last a lifetime,” Bennet said on the Senate floor. “At the same time, breakthrough treatments have become available for adults with better results and few harmful effects. While these treatments have great promise for kids, we’re not doing enough to explore that potential.”

Advocates cheered the bill’s passage.

“Today's Senate vote is a giant leap forward in the fight against childhood cancer,.” said Jorge Luis Lopez, a Miami attorney who sits on the board of the American Cancer Society. "We urge Donald Trump to sign this legislation into law and unleash American innovation and creativity for the health and well-being of all our children.”

On Wednesday, Rubio was on the Senate floor sharing a story about Bella Rodriguez-Torres, the 10-year-old girl from Miami whose 2013 death helped get him involved.

“She was a classmate of my nephew in grade school,” Rubio said. “And she lost her battle with cancer. Her father has been a tireless advocate on this cause. He moved heaven and earth to try to reach a point where they could find a cure for her. That did not come in time and he’s now made it the mission of his life to honor her life by continuing this work. So we’ve all been impacted in some way."